PharmaRadar360

PharmaRadar360

NeurologyDrug intelligence

Isembyld

Apitegromab

Antibody that blocks myostatin activation (binds promyostatin and latent myostatin). Developed by Scholar Rock. Clinical, regulatory, and source evidence are connected below.

Company
Scholar Rock
Target / MOA
Myostatin 路 MSTN
Aliases
Isembyld
Signals
7
Clinical trials
6
Approvals
0
Evidence sources
7

Latest signals

What changed

The three most recent source-backed changes linked to this drug.

clinical trialClinicalTrials.gov

A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy

Phase 2 路 RECRUITING 路 Scholar Rock, Inc.

clinical trialClinicalTrials.gov

Phase 2 Study Evaluating Apitegromab for the Treatment of FSHD

Phase 2 路 RECRUITING 路 Scholar Rock, Inc.

clinical trialClinicalTrials.gov

Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab

Phase 3 路 ACTIVE_NOT_RECRUITING 路 Scholar Rock, Inc.

Approved uses

What regulators have approved

The indications section of each current US label, word for word, and the EU summary of each authorised product. Official sources as of Oct 21, 2026.

United States (FDA label)

Isembyld 路 BLA761463 路 label version 3, effective Sep 14, 2026 路 DailyMed

Label section 1

1 INDICATIONS AND USAGE ISEMBYLD is indicated for the treatment of spinal muscular atrophy (SMA) in adults and pediatric patients 2 years of age and older who are currently receiving a survival motor neuron 2 (SMN2)-targeted treatment.

European Union (EMA)

EMA's medicines data lists no marketing authorisation for this medicine.

Not covered here: Isembyld's EU application (EMEA/H/C/005909) (Withdrawn, per EMA's medicines data.)

Regulatory history

Approvals over time

US original approvals and efficacy supplements (new indications, populations or dosing) from Drugs@FDA; EU authorisations and CHMP opinions on extensions. Official sources as of Oct 21, 2026.

United States

BLA761463 路 SCHOLAR ROCK INC 路 first approved Sep 11, 2026

European Union

EMA's medicines data lists no marketing authorisation for this medicine.

    Patents and exclusivity

    What FDA lists (US)

    Patents and exclusivity as FDA lists them in the Orange Book (drugs) and the Purple Book (biologics), with approved generics and biosimilars. Expired listings are not shown. Official sources as of Oct 21, 2026.

    BLA761463 (Purple Book)

    No exclusivity date listed.

    Generics and biosimilars

    The Purple Book lists no licensed biosimilar.

    FDA's listings only: an approved generic or licensed biosimilar may not be on the market yet. Not a forecast of when they may launch, and not legal advice.

    Safety

    Boxed warnings and FDA safety communications

    The boxed warning on each current US label, and FDA Drug Safety Communications that name the drug (FDA's list from January 2020). Official sources as of Oct 21, 2026.

    Boxed warning

    The current US label has no boxed warning.

    FDA Drug Safety Communications

    None on FDA's list names this drug.

    Clinical trials

    Key active studies

    A focused view of recently updated studies 路 6 linked records.

    Phase 2RECRUITINGNCT07047144

    A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy

    Scholar Rock, Inc. 路 Updated Oct 2, 2026

    Phase 2RECRUITINGNCT07435129

    Phase 2 Study Evaluating Apitegromab for the Treatment of FSHD

    Scholar Rock, Inc. 路 Updated Sep 2, 2026

    Phase 3ACTIVE NOT RECRUITINGNCT05626855

    Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab

    Scholar Rock, Inc. 路 Updated Sep 1, 2026

    N/ANO LONGER AVAILABLENCT06877689

    EAP of Apitegromab for Patients With Spinal Muscular Atrophy

    Scholar Rock, Inc. 路 Updated Apr 28, 2026

    Phase 2COMPLETEDNCT06445075

    Efficacy and Safety of Apitegromab for the Treatment of Adults Who Are Overweight or Obese

    Scholar Rock, Inc. 路 Updated Jan 23, 2026

    Phase 3COMPLETEDNCT05156320

    Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam

    Scholar Rock, Inc. 路 Updated Jan 22, 2026

    Regulatory timeline

    Approvals and agency activity

    Verified regulatory records in reverse chronological order.

    No regulatory event is available in this snapshot.

    Source evidence

    Where this profile comes from

    Facts link back to original registries, regulators, labels, and publishers. Counts change as source records update.

    ClinicalTrials.govofficial 路 Latest Oct 2, 20267
    Professional referenceThis profile summarizes linked public evidence. It is not medical, legal, regulatory, or investment advice. Verify decisions against original sources.